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May 15, 2011 (Vol. 31, No. 10)


  • Resources for patients and medical professionals
  • Unable to simply browse some databases

It’s often a hard-knock life for researchers looking to develop orphan drugs. These drugs are specifically formulated to treat rare medical conditions, which means that there is not much of a market for them, speaking in a strictly dollars-and-cents manner. Researchers, doctors, patients, and family members are well aware of the obstacles impeding progress in the medical care of rare diseases, and Orphanet exists to help overcome those impedances. With the goal of contributing “to the improvement of the diagnosis, care, and treatment of patients with rare diseases,” Orphanet is a database containing information on rare diseases, orphan drugs, expert centers, diagnostic tests, and research/clinical trials. Beyond simply providing scientific information for researchers and caregivers, there are also resources for patients, such as a directory of patient organizations.

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*The opinions expressed are solely those of the author(s) and should not be construed as reflecting the viewpoints of the publisher, Genetic Engineering & Biotechnology News, Mary Ann Liebert, Inc., the publishing house, or employees and affiliates thereof.

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